Is Zolgensma suitable for children and adults?
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Zolgensma
Zolgensma has been mostly tested in infants and toddlers with spinal muscular atrophy, in whom it was deemed safe and able to improve or maintain motor function. A couple of trials also have enrolled adolescents as old as age 17 but the gene therapy has never been tested in adults, who tend to have milder forms of the disease. In the U.S., the therapy is only approved for children in the first two years of life.
